Modified CRISPR technique treats diseases without cutting DNA

New York, Dec 8 (IANS) In what could remove a major hurdle to using gene editing technologies to treat human diseases, scientists have created a new version of the CRISPR/Cas9 genome editing technology that allows them to activate genes and treat diseases without creating breaks in the DNA.

Most CRISPR/Cas9 systems work by creating “double-strand breaks” (DSBs) in regions of the genome targeted for editing or for deletion.

But many researchers are opposed to creating such breaks in the DNA of living humans because there are growing concerns regarding unwanted mutations generated by the breaks.

“Cutting DNA opens the door to introducing new mutations,” said Juan Carlos Izpisua Belmonte of the Salk Institute for Biological Studies whose laboratory developed the new technique.

“That is something that is going to stay with us with CRISPR or any other tool we develop that cuts DNA. It is a major bottleneck in the field of genetics — the possibility that the cell, after the DNA is cut, may introduce harmful mistakes,” he said.

The new technique, detailed in the journal Cell, alters the activity, rather than the underlying sequence, of disease-associated genes.

As a proof of concept, the researchers used their new approach to treat several diseases, including diabetes, acute kidney disease and muscular dystrophy, in mouse models.

The researchers developed the technique using a modified CRISPR-Cas9 system that does not cut the DNA.